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公开(公告)号：EP3102673A4
公开(公告)日：2017-11-01
申请号：EP15742584
申请日：2015-02-02
Applicant: SANGAMO BIOSCIENCES INC
Inventor： LIU PEI-QI , REIK ANDREAS , ZHANG LEI
CPC classification number: C07K14/805 , A61K35/28 , A61K2035/124
Abstract: Methods and compositions for treatment of a beta thalessemia are provided.
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公开(公告)号：EP3201339A2
公开(公告)日：2017-08-09
申请号：EP15846242.4
申请日：2015-10-03
Applicant: Cold Spring Harbor Laboratory
Inventor： KRAINER, Adrian , AZNAREZ, Isabel
IPC: C12N15/113
CPC classification number: C12N15/1137 , C07K14/47 , C07K14/4702 , C07K14/4736 , C07K14/805 , C12N15/111 , C12N15/113 , C12N2310/11 , C12N2310/315 , C12N2310/3341 , C12N2320/11 , C12N2320/33 , C12Q1/6883 , C12Q2600/136 , C12Q2600/158 , C12Y101/01205 , C12Y304/24087 , C12N2310/321 , C12N2310/3521
Abstract: Provided herein are methods and compositions for increasing production of a target protein or functional RNA by a cell.
Abstract translation: 本文提供了用于增加细胞产生靶蛋白或功能性RNA的方法和组合物。
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公开(公告)号：EP3189143A1
公开(公告)日：2017-07-12
申请号：EP15837740.8
申请日：2015-09-04
Inventor： SADELAIN, Michel , RIVIERE, Isabelle , MANSILLA-SOTO, Jorge , WANG, Xiuyan , STAMATOYANNOPOULOS, George , STAMATOYANNOPOULOS, John , LIU, Mingdong
IPC: C12N15/113
CPC classification number: A61K48/0066 , A61K48/0058 , C07K14/805 , C12N9/22 , C12N15/85 , C12N15/86 , C12N2740/10043 , C12N2740/15043 , C12N2740/16043 , C12N2830/008 , C12N2830/15 , C12N2830/30 , C12N2830/40 , C12N2830/46 , C12N2830/48 , C12N2830/50
Abstract: The presently disclosed subject matter provides for expression cassettes that allow for expression of a globin gene or a functional portion thereof, vectors comprising thereof, and cells transduced with such expression cassettes and vectors. The presently disclosed subject matter further provides methods for treating a hemoglobinopathy in a subject comprising administering an effective amount of such transduced cells to the subject.
Abstract translation: 本发明公开的主题提供了允许表达珠蛋白基因或其功能部分的表达盒，包含其的载体以及用这种表达盒和载体转导的细胞。 本发明公开的主题进一步提供了用于治疗受试者中的血红蛋白病的方法，其包括向受试者施用有效量的这样的转导细胞。
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公开(公告)号：EP2334794B8
公开(公告)日：2017-04-19
申请号：EP09813730.0
申请日：2009-09-14
Inventor： ORKIN, Stuart, H. , SANKARAN, Vijay, G.
IPC: C12N15/63 , C12N15/113 , A61K31/7088
CPC classification number: C12N15/113 , A01K67/0275 , A01K2217/052 , A01K2217/15 , A01K2227/105 , A01K2267/0381 , A61K31/713 , A61K35/28 , A61K49/00 , C07K14/4705 , C07K14/805 , C12N5/0647 , C12N15/8509 , C12N15/86 , C12N2310/14 , C12N2310/531 , C12N2740/15043 , C12N2800/206
Abstract: The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies.
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公开(公告)号：EP2890780A4
公开(公告)日：2016-08-31
申请号：EP13833658
申请日：2013-08-29
Applicant: SANGAMO BIOSCIENCES INC
Inventor： COST GREGORY J , GREGORY PHILIP D , GUSCHIN DMITRY , HOLMES MICHAEL C , MILLER JEFFREY C , PASCHON DAVID , REBAR EDWARD J , REIK ANDREAS , URNOV FYODOR , ZHANG LEI
IPC: C12N5/071 , C07K14/47 , C07K14/805 , C12N9/22 , C12N15/85
CPC classification number: C12N15/85 , C07K14/4702 , C07K14/805 , C07K2319/81 , C12N9/22 , C12N15/52
Abstract: Disclosed herein are nucleases and methods of using these nucleases for alteration of a globin gene and generation of cells.
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Patent Agency Ranking6.发明公开
公开(公告)号：EP2895611A4
公开(公告)日：2016-04-27
申请号：EP13836738
申请日：2013-09-10
Applicant: UNIV CALIFORNIA
Inventor： COOPER AARON , KOHN DONALD B , URBINATI FABRIZIA , GARCIA ZULEMA R , HOLLIS ROGER , GEIGER-SCHREDELSEKER SABINE , SENADHEERA SHANTHA
IPC: C12N15/867 , A61K48/00 , C07K14/805
CPC classification number: A61K48/0066 , C07K14/805 , C12N15/86 , C12N2740/16043 , C12N2830/40 , C12N2830/46 , C12N2830/48
Abstract: In various embodiments a recombinant lentiviral vector is provided comprising an expression cassette comprising a nucleic acid construct comprising an anti-sickling human beta globin gene encoding an anti-sickling-beta globin polypeptide comprising the mutations Gly16Asp, Glu22Ala and Thr87Gln, where the lentiviral vector is a TAT-independent and self-inactivating (SIN). In certain embodiments the vector additionally contains one or more insulator elements. The vectors are useful in gene therapy for the treatment of sickle cell disease.
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公开(公告)号：EP2830638A4
公开(公告)日：2015-12-16
申请号：EP13769757
申请日：2013-03-15
Applicant: SCHINDLER WILLIAM
Inventor： VANDEGRIFF KIM D , MALAVALLI ASHOK , OLSEN SCOTT D
IPC: A61K35/14
CPC classification number: A61K38/42 , A61K47/48215 , A61K47/60 , C07K1/10 , C07K14/805 , C12N9/0044
Abstract: PEGylated diaspirin-crosslinked hemoglobin conjugates having high oxygen affinity are described which have enhanced nitrite reductase activity to deliver oxygen, carbon monoxide, nitric oxide or mixtures thereof to tissues to treat various diseases and conditions.
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8.发明公开LENTIVIRAL VECTOR FOR STEM CELL GENE THERAPY OF SICKLE CELL DISEASE 审中-公开LENTIVIRALE VEKTOREN ZUR STAMMZELLENGENTHERAPIE VONSICHELZELLANÄMIE
公开(公告)号：EP2895611A1
公开(公告)日：2015-07-22
申请号：EP13836738.8
申请日：2013-09-10
Applicant: The Regents of the University of California
Inventor： COOPER, Aaron , KOHN, Donald B. , URBINATI, Fabrizia , GARCIA, Zulema R. , HOLLIS, Roger , GEIGER-SCHREDELSEKER, Sabine , SENADHEERA, Shantha
IPC: C12N15/867 , C12N15/12 , A61K48/00
CPC classification number: A61K48/0066 , C07K14/805 , C12N15/86 , C12N2740/16043 , C12N2830/40 , C12N2830/46 , C12N2830/48
Abstract: In various embodiments a recombinant lentiviral vector is provided comprising an expression cassette comprising a nucleic acid construct comprising an anti-sickling human beta globin gene encoding an anti-sickling-beta globin polypeptide comprising the mutations Gly16Asp, Glu22Ala and Thr87Gln, where the lentiviral vector is a TAT-independent and self-inactivating (SIN). In certain embodiments the vector additionally contains one or more insulator elements. The vectors are useful in gene therapy for the treatment of sickle cell disease.
Abstract translation: 在各种实施方案中，提供重组慢病毒载体，其包含包含核酸构建体的核酸构建体，所述核酸构建体包含编码包含突变Gly16Asp，Glu22Ala和Thr87Gln的抗镰状蛋白β球蛋白多肽的抗镰状人β球蛋白基因，其中慢病毒载体为 一个独立于TAT的自动灭活（SIN）。 在某些实施方案中，载体还包含一个或多个绝缘体元件。 载体可用于治疗镰状细胞病的基因治疗。
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9.发明公开SUCCINIMIDE-ACTIVATED NITROXYL COMPOUNDS AND METHODS FOR THE USE THEREOF FOR NITROXYLATION OF PROTEINS 审中-公开SUCCINIMIDAKTIVIERTE硝和方法及其用于蛋白质dinitroxylation
公开(公告)号：EP2834329A1
公开(公告)日：2015-02-11
申请号：EP13771756.7
申请日：2013-03-15
Applicant: Sangart, Inc.
Inventor： VANDEGRIFF, Kim D. , MALAVALLI, Ashok , MKRTCHYAN, Gnel
IPC: C10L1/22
CPC classification number: C07K14/805 , A61K38/42 , C07D207/416 , C07D401/12 , C07D401/14 , C07D403/12 , C07K14/765
Abstract: The present invention relates to succinimide-activated nitroxyl compounds and methods for the synthesis of such compounds. The present invention also relates to the use of succinimide-activated nitroxyl compounds to prepare nitroxylated proteins, for example nitroxylated heme proteins (e.g., nitroxylated hemoglobin and nitroxylated myoglobin). The nitroxylated proteins are optionally also conjugated to a polyalkylene oxide (PAO), for example to a polyethylene glycol (PEG). Polynitroxylated heme proteins are useful as oxygen therapeutic agents (OTAs). The invention further relates to pharmaceutical compositions of the nitroxylated proteins and methods for the use of nitroxylated proteins in the treatment of various conditions.
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公开(公告)号：EP2094292B1
公开(公告)日：2014-07-09
申请号：EP07843765.4
申请日：2007-10-03
Applicant: Ikor, Inc.
Inventor： TYE, Ross Walden
IPC: A61K38/42 , C07K14/805
CPC classification number: A61K38/42 , C07K14/805 , Y02P20/55
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